Primary hyperoxaluria type 1 (PH1) leads to end stage renal disease (ESRD) and systemic manifestations. Phenotypic variability is the norm among PH1 patients and timing of ESRD is hard to predict. Even though liver transplantation is currently the only definitive cure, the utilization of preemptive liver transplantation (PLT), while kidney function is still normal, is not trivial. The ideal timing of PLT as well as its effects on preservation of renal function are unknown.

Objective: To determine the effects of PLT on preservation of renal function in patients with PH1.

Material and methods:

A retrospective, medical record based, single center trial of PH1 patients who were treated in our center between 1997 -2017.

Results:

36 PH1 patients were enrolled in this study. Among them, 18 presented with end stage renal disease (ESRD group). From the 18 patients who were eligible for PLT (ePLT group) (eGFR > 40 mil/min/ 1.73m2 at diagnosis), only seven patients chose to undergo PLT (PLT group) while 11 continued conservative treatments (nPLT group). In the PLT group, the median eGFR at presentation and at the time of PLT was 77 mil/min/1.73m2(range 50.7-106) and 72 mil/min/1.73m2 (50-89) respectively. Over time, the renal function improved in the PLT group and at the end of the follow up period (16-20 years), median eGFR was 104 mil/min/1.73m2 (86-108). During the follow up period no patient died, reached ESRD or dialysis in the PLT group. In the nPLT group the median age of dialysis start was 20.7 years and the median time to first transplantation was 12.75 years. The mortality rates were significantly higher in the ESRD and the nPLT groups; 11 (61%) and 2 (18%) respectively, p<0.05. Perioperative mortality occurred only in the ESRD (3) and nPLT (1) groups.

Conclusions:

Early preemptive liver transplantation preserves renal function and might prevent mortality in patients with PH1. However, the morbidity and mortality of liver transplantation should be considered carefully when the candidate has normal renal function and the timing of ESRD cannot be determined.